SMA REACH UK is a two year study funded by a generous grant from SMA UK to Great Ormond Street Children's Charity for which we are very grateful. The SMA REACH UK project is a new initiative in collaboration with existing UK SMA registries - the UK SMA Patient Registry AND SMArtNet Clinical Network UK sponsored by Muscular Dystrophy UK and Spinal Muscular Atrophy Support UK (formerly The Jennifer Trust). The SMA REACH UK project is led by the study team based at the Dubowitz Neuromuscular Centre, UCL
The primary aim of this project is to establish the first national clinical and research network: SMA REACH UK - SMA Research And Clinical Hub UK - to promote a national agreement on clinical and physiotherapy assessment and standards of care. We plan to design, pilot and expand an electronic database created to streamline the collection of data for patients with SMA. This UK SMA database would be a unique infrastructure started at GOSH and Newcastle which would soon be accessible to specialist centres across the UK who treat patients with SMA.
The secondary aim of the project is to utilize the SMA REACH UK database as a longitudinal data store where information can be audited and reviewed. This will provide clinicians and researchers a rich resource of available information on a large collection of SMA patients.
We are collaborating with the Catholic University Rome, an International centre of excellence in SMA research and treatment, with the shared goal to facilitate translational research for this common neuromuscular disease in preparation to design National and International clinical trials.
Once the system is finalised, additional national sites that have a history of successful SMA enrolment will be invited to participate and collect high quality longitudinal data.
This work will be an invaluable tool for the centres likely to be involved in upcoming SMA multicentre randomised clinical trials in SMA type I, II and III. We also aim to ensure that the functional scales used are suitable and clinically relevant for future trials.
The primary aim of this project is to establish the first national clinical and research network: SMA REACH UK - SMA Research And Clinical Hub UK - to promote a national agreement on clinical and physiotherapy assessment and standards of care. We plan to design, pilot and expand an electronic database created to streamline the collection of data for patients with SMA. This UK SMA database would be a unique infrastructure started at GOSH and Newcastle which would soon be accessible to specialist centres across the UK who treat patients with SMA.
The secondary aim of the project is to utilize the SMA REACH UK database as a longitudinal data store where information can be audited and reviewed. This will provide clinicians and researchers a rich resource of available information on a large collection of SMA patients.
We are collaborating with the Catholic University Rome, an International centre of excellence in SMA research and treatment, with the shared goal to facilitate translational research for this common neuromuscular disease in preparation to design National and International clinical trials.
Once the system is finalised, additional national sites that have a history of successful SMA enrolment will be invited to participate and collect high quality longitudinal data.
This work will be an invaluable tool for the centres likely to be involved in upcoming SMA multicentre randomised clinical trials in SMA type I, II and III. We also aim to ensure that the functional scales used are suitable and clinically relevant for future trials.
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